Targeted nonviral delivery of genome editors in vivo

CA Tsuchida, KM Wasko… - Proceedings of the …, 2024 - National Acad Sciences
Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that
will drive biological discovery and transform the field of cell and gene therapy. Here, we …

Enhanced local delivery of Engineered IL-2 mRNA by porous silica nanoparticles to promote effective Antitumor immunity

H Shin, S Kang, C Won, DH Min - ACS nano, 2023 - ACS Publications
Localized expression of immunomodulatory molecules can stimulate immune responses
against tumors in the tumor microenvironment while avoiding toxicities associated with …

Extracellular vesicle‐mediated delivery of CRISPR/Cas9 ribonucleoprotein complex targeting proprotein convertase subtilisin‐kexin type 9 (Pcsk9) in primary mouse …

NF Ilahibaks, TA Kluiver, OG de Jong… - Journal of …, 2024 - Wiley Online Library
The loss‐of‐function of the proprotein convertase subtilisin–kexin type 9 (Pcsk9) gene has
been associated with significant reductions in plasma serum low‐density lipoprotein …

Non-viral delivery of nucleic acid for treatment of rare diseases of the muscle

D Rao, M Ganguli - Journal of Biosciences, 2024 - Springer
Rare muscular disorders (RMDs) are disorders that affect a small percentage of the
population. The disorders which are attributed to genetic mutations often manifest in the form …

Confinement in Dual-Chain-Locked DNA Origami Nanocages Programs Marker-Responsive Delivery of CRISPR/Cas9 Ribonucleoproteins

Z Xu, Y Dong, N Ma, X Zhu, X Zhang… - Journal of the …, 2023 - ACS Publications
Delivery of CRISPR/Cas9 ribonucleoproteins (RNPs) offers a powerful tool for therapeutic
genome editing. However, precise manipulation of CRISPR/Cas9 RNPs to switch the …

Development of a cancer nanovaccine to induce antigen-specific immune responses based on large-sized Porous silica nanoparticles

H Shin, S Kang, SY Chae, C Won… - ACS Applied Materials & …, 2023 - ACS Publications
Cancer vaccine is one of the immunotherapeutic strategies aiming to effectively deliver
cancer antigens to professional antigen-presenting cells such as dendritic cells (DCs) …

Enhancement of gene editing and base editing with therapeutic ribonucleoproteins through in vivo delivery based on absorptive silica nanoconstruct

S Kim, YK Jeong, CS Cho, SH Lee… - Advanced …, 2023 - Wiley Online Library
Key to the widespread and secure application of genome editing tools is the safe and
effective delivery of multiple components of ribonucleoproteins (RNPs) into single cells …

Metabolic Modulation of Kynurenine Based on Kynureninase-Loaded Nanoparticle Depot Overcomes Tumor Immune Evasion in Cancer Immunotherapy

SY Chae, H Shin, J Woo, S Kang… - ACS Applied Materials …, 2024 - ACS Publications
Evading recognition of immune cells is a well-known strategy of tumors used for their
survival. One of the immune evasion mechanisms is the synthesis of kynurenine (KYN), a …

A quaternary ammonium-based nanosystem enables delivery of CRISPR/Cas9 for cancer therapy

M Zhang, S Sun, X Liang, Z Liu, J Yin, Q Li… - Biomaterials …, 2024 - pubs.rsc.org
Genome editing mediated by CRISPR/Cas9 is an attractive weapon for cancer therapy.
However, in vivo delivery of CRISPR/Cas9 components to achieve therapeutic efficiency is …

Nanotechnology‐based CRISPR/Cas9 delivery system for genome editing in cancer treatment

S Zhou, Y Li, Q Wu, C Gong - MedComm–Biomaterials and …, 2024 - Wiley Online Library
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR
associated protein 9 (CRISPR/Cas9) systems initiate a revolution in genome editing, which …